Mulpleta (lusutrombopag) / Shionogi - LARVOL DELTA

Repurposing simeprevir uncovers a druggable KPNB1-p65-BCL2 survival axis in cancer. (PubMed, Cell Chem Biol) - "Through screening of an FDA-approved compound library, we uncovered simeprevir (Sim) and lusutrombopag (Lus) as direct KPNB1 inhibitors that disrupt its import complex assembly. Analysis of The Cancer Genome Atlas (TCGA) datasets substantiated the clinical relevance of the KPNB1-p65-Bcl-2 axis. Our study elucidates a therapeutically targetable KPNB1-p65-Bcl-2 survival axis in multiple cancers and establishes a foundation for developing next-generation KPNB1 inhibitors."

IO biomarker • Journal • Oncology • BCL2 • KPNB1 • RELA

Platelet optimization in high-risk ALBI grade 3 patients undergoing invasive procedures: real-world evidence from lusutrombopag therapy. (PubMed, Front Pharmacol) - "These findings are exploratory and should not be interpreted as evidence of equivalent efficacy or established safety across ALBI strata. Larger prospective studies are needed to validate these results."

HEOR • Journal • Real-world evidence • Hematological Disorders • Hepatocellular Cancer • Oncology • Solid Tumor • Thrombocytopenia

EFFICACY AND SAFETY OF LUSUTROMBOPAG IN PATIENTS WITH NON-SEVERE APLASTIC ANEMIA: RESULTS FROM A PROSPECTIVE, SINGLE-ARM, OPEN-LABEL, EXPLORATORY STUDY (EHA 2026) - P4 | "The drug was well-tolerated with no treatment-related adverse events reported. These findings support Lusutrombopag is a potential therapeutic option for NSAA and warrant further investigation in larger, randomized controlled trials."

Clinical • Aplastic Anemia • Hematological Disorders • Pancreatitis

EFFICACY AND SAFETY OF HETROMBOPAG IN INCREASING PLATELET COUNTS IN PATIENTS WITH HEPATOCELLULAR CARCINOMA UNDERGOING HEPATECTOMY: A RANDOMIZED, PLACEBO-CONTROLLED STUDY (EHA 2026) - P2 | "Similar agents like avatrombopag and lusutrombopag have been approved for TCP in chronic liver disease patients undergoing a procedure. Hetrombopag represents an effective, non ‑ invasive alternative to perioperative platelet transfusions in this population. Trial registration: NCT05442632."

Clinical • Aplastic Anemia • Hematological Disorders • Hepatocellular Cancer • Hepatology • Immune Thrombocytopenic Purpura • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura

EFFICACY AND SAFETY OF LUSUTROMBOPAG IN PERSISTENT OR CHRONIC PRIMARY IMMUNE THROMBOCYTOPENIA: A PROSPECTIVE, OPEN-LABEL, SINGLE-ARM PHASE II STUDY (EHA 2026) - P2 | "Summary/Conclusion In Chinese adult patients with persistent or chronic primary ITP, lusutrombopag provides rapid and sustained platelet response with a favorable safety profile. The study suggests that lusutrombopag, with a dose-titration strategy, is a promising therapeutic option for primary ITP, warranting further validation in large-scale randomized controlled trials."

Clinical • P2 data • Cardiovascular • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Thrombopoietin receptor agonists in immune thrombocytopenia: a comparative review of mechanisms, efficacy, and the future of personalized management. (PubMed, Expert Opin Pharmacother) - "The three approved agents - romiplostim, eltrombopag, and avatrombopag - all stimulate the c-Mpl receptor but differ in their molecular interactions, pharmacokinetics, and potential immunomodulatory properties...TPO-RAs now represent a cornerstone of chronic ITP management and are expected to see broader use as growing evidence supports earlier and more individualized treatment strategies. Future progress will rely on personalized therapeutic strategies, informed monitoring, and AI-driven predictive tools that together may enhance remission durability and long-term clinical outcomes."

Journal • Review • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and Safety of Lusutrombopag After Allogeneic Hematopoietic Stem Cell Transplantation: a Single-arm, Open-label, Prospective Clinical Study (clinicaltrials.gov) - P=N/A | N=45 | Recruiting | Sponsor: The General Hospital of Western Theater Command

New trial • Bone Marrow Transplantation • Hematological Disorders • Transplantation

Repurposing lusutrombopag: unveiling anti-Staphylococcus aureus activity in a novel oral thrombopoietin receptor agonist. (PubMed, BMC Microbiol) - No abstract available

Journal

Lusutrombopag in the Treatment of Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P2 | N=17 | Completed | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Recruiting ➔ Completed | Trial completion date: Nov 2024 ➔ Mar 2025

Trial completion • Trial completion date • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Lusutrombopag improves platelet engraftment after autologous haematopoietic stem cell transplantation in multiple myeloma: A single-centre retrospective study (ASH 2025) - "Result Lusutrombopag accelerated platelet engraftment: Median engraftment time: 10 days (lusutrombopag) vs 12 days (controls) Cumulative incidence at day +13: Significantly higher in lusutrombopag group (P*=0.002; 95% CI: -3.5 to -1.0 days) ≤10-day engraftment rate: Significantly increased (OR=12.5; P*=0.006) Both groups exhibited favourable outcomes with no adverse events. Conclusion Lusutrombopag accelerates platelet engraftment post-ASCT in MM patients, reducing median time by 2 days and transfusion requirements, thereby optimising this critical treatment phase."

Retrospective data • Hematological Malignancies • Multiple Myeloma • Transplantation

FLT3 ligand facilitates long-term ex vivo expansion of human hematopoietic stem cells by maintaining lymphoid reconstitution potential (ASH 2025) - "In a short-term (7-day) culture, we foundthat supplementing SCF in addition to 740 Y-P, or substituting butyzamide with lusutrombopag, an FDA-approved TPO-RA, enhanced the ex vivo expansion of phenotypic hematopoietic stem and progenitorcells (lineage⁻CD41⁻CD34⁺) from CD34+ umbilical cord blood (UCB) cells...In contrast, the 5LS culture demonstrated greater fold expansion of functional HSCs after 14 days(60.6-fold expansion) compared to the 5LSF culture (23.1-fold expansion), which is consistent with theresults from the phenotypic characterization. These findings provide evidence that human functional HSCcan stably expands long-term in the 5LSF culture.In conclusion, we have developed a long-term expansion protocol for human HSCs that facilitates bothclinical and research applications of these functional stem cells."

Preclinical • Hematological Disorders • Inflammation • CD33 • CD34 • FLT3 • ITGA2B • ITGA3 • PROCR • STAT5 • THY1

Prolonged thrombocytopenia following diverse CAR-T cell therapies: High incidence, inflammatory risk factors, and a comparative efficacy analysis of TPO-receptor agonist strategies in a real-world cohort (ASH 2025) - "The efficacy of TPO-RA interventions—includingmonotherapy (Eltrombopag, Avatrombopag, Hetrombopag, Lusutrombopag, Romiplostim, or rhTPO) andcombination strategies—was evaluated. Prolonged, severe thrombocytopenia is a frequent, inflammation-driven complication of CAR-T therapy. Our real-world data demonstrate a clear efficacy hierarchy among TPO-RAs, with romiplostimbeing the most effective single agent. Crucially, combination TPO-RA strategies, particularly thoseinvolving romiplostim or sequential rhTPO, significantly accelerate platelet recovery in severe caseswithout increasing toxicity."

CAR T-Cell Therapy • Clinical • Real-world • Real-world evidence • B Cell Non-Hodgkin Lymphoma • Cardiovascular • Hematological Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • IFNG

Efficacy and safety of lusutrombopag monotherapy for cyclosporine A-refractory, transfusion-dependent non-severe aplastic anemia (PubMed, Zhonghua Xue Ye Xue Za Zhi) - "During follow-up, one patient experienced a loss of OR after discontinuing treatment, with a relapse rate of 14.3%; no clonal evolution or mortality was observed. These findings suggest that lusutrombopag is both effective and well-tolerated in CsA-refractory TD-NSAA patients and represents a promising therapeutic option for those with poor treatment tolerability."

Journal • Monotherapy • Retrospective data • Anemia • Aplastic Anemia • Hematological Disorders

Lusutrombopag or hetrombopag supports in vitro megakaryopoiesis better than other thrombopoietin receptor agonists. (PubMed, Br J Haematol) - No abstract available

Journal • Preclinical

A Prospective Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of Lusutrombopag in Chinese Adults with Persistent or Chronic Primary Immune Thrombocytopenia (ASH 2024) - P2 | "TPO-RAs (romiplostim, eltrombopag, hetrombopag, and avatrombopag) has been approved for treating ITP in Chinese...Prior to enrollment, 50% had been receiving stable steroids (same milligram amount ±10% and ≤ 20mg of equivalent dose of prednisone) for ≥2 weeks and no other concomitant medications affecting PLTs were administered...Treatment emergent adverse events (TEAEs) occurred in four of eight patients and there were no treatment-related adverse reactions. None experienced serious adverse events (SAE).Conclusions : Lusutrombopag, a newly approved TPO-RA in China, has shown preliminary efficacy initiated with a once-daily 3mg tablet with tolerability in Chinese adults with persistent or chronic primary ITP."

Clinical • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Development and validation of a prognostic prediction model based on coagulation-related genes and clinical factors in acute leukemia. (PubMed, Transl Pediatr) - "The model helps to enable individualised risk stratification and guide targeted therapy. In addition, avatrombopag and lusutrombopag as potential therapeutic agents provide new ideas for precision medicine in paediatric AL."

Journal • Hematological Malignancies • Leukemia • Oncology • Pediatrics • PROS1

Relationship between thromboembolic events and thrombopoietin receptor agonists: a pharmacovigilance analysis of the FDA Adverse Event Reporting System and the Japanese Adverse Drug Event Report. (PubMed, BMJ Open) - "The study provided real-world evidence of TEE associated with TPO-RAs, highlighting a strong link despite variations in signal values and regional reporting practices. Findings underscore ongoing clinical safety surveillance for TPO-RAs."

Adverse events • Journal • Retrospective data • Cardiovascular • Hematological Disorders • Thrombocytopenia

An open-lable, single-arm pilot study to evaluate the efficacy and safety of lusutrombopag in Chinese adults with persistent or chronic immune thrombocytopenia (ChiCTR) - P4 | N=17 | Completed | Sponsor: Institute of Hematology& Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; Institute of Hematology&a | Recruiting ➔ Completed

Trial completion • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and safety of lusutrombopag in the treatment of patients with non-severe aplastic anemia (NSAA). (ChiCTR) - P4 | N=15 | Recruiting | Sponsor: Tianjin Medical University General Hospital; Tianjin Medical University General Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Anemia • Aplastic Anemia • Hematological Disorders

Pharmacist intervention through protocol-based pharmacotherapy management is effective to ensure safety in invasive procedures for chronic liver disease. (PubMed, J Pharm Health Care Sci) - "The use of platelet products decreases without the increased incidences of hemorrhage if pharmacists suggest prescribing thrombopoietin receptor agonists based on their assessment of the platelet count and the bleeding risk of the procedure."

Journal • Hematological Disorders • Hepatology • Thrombocytopenia

Lusutrombopag combined with recombinant human thrombopoietin for the treatment of thrombocytopenia in patients with chronic liver disease destined to undergo elective invasive surgery: a prospective, multicentre, single-arm clinical study (ChiCTR) - P2 | N=60 | Not yet recruiting | Sponsor: THE FIRST AFFILIATED HOSPITAL OF USTC (ANHUI PROVINCAL HOSPITAL); THE FIRST AFFILIATED HOSPITAL OF USTCANHUI PROVINCAL HOSPITAL

New P2 trial • Hematological Disorders • Hepatology • Thrombocytopenia

Usefulness of Repeated Lusutrombopag Administration in Clinical Practice and its Correlation with the Simulation Software-Predicted Platelet Count: A Multicenter Study. (PubMed, Intern Med) - "There was a correlation between the highest software-predicted platelet count and the platelet count measured in actual clinical practice in all dose frequency groups; however, the correlation was the strongest in the one-dose group (r = 0.74, p <0.0001). Conclusion Frequent lusutrombopag administration is therefore both effective and safe, and a platelet prediction simulation software program is thus considered to be useful in practice."

Clinical • Journal • Hematological Disorders • Hepatology • Immunology • Liver Cancer • Oncology • Solid Tumor • Thrombocytopenia

QSAR-Based Drug Repurposing and RNA-Seq Metabolic Networks Highlight Treatment Opportunities for Hepatocellular Carcinoma Through Pyrimidine Starvation. (PubMed, Cancers (Basel)) - "For DHODH, the drugs Oteseconazole, Tipranavir, and Lusutrombopag were identified as potential inhibitors. For TYMS, the drugs Tadalafil, Dabigatran, Baloxavir Marboxil, and Candesartan Cilexetil showed promise as inhibitors. Overall, this study suggests in vitro testing of the identified drugs to assess their capabilities in inducing pyrimidine starvation on HCC."

Journal • Hepatocellular Cancer • Oncology • Solid Tumor • TYMS

Clinical research progress of lusutrombopag for the treatment of thrombocytopenia in chronic liver disease (PubMed, Zhonghua Gan Zang Bing Za Zhi) - "Lusutrombopag is a newly developed small-molecule TPO receptor agonist that can induce bone marrow progenitor cells to differentiate into megakaryocytes and increase platelet production, posing characteristics of oral convenience, safety and effectiveness; thus, it has been used in many countries and regions for the treatment of patients with chronic liver diseases concurrent with thrombocytopenia. Herein, the pharmacological features and clinical research are reviewed."

Journal • Review • Hematological Disorders • Hepatology • Thrombocytopenia

Lusutrombopag Combined With Recombinant Human Thrombopoietin for the Treatment of Thrombocytopenia in Patients With Chronic Liver Disease Destined to Undergo Elective Invasive Surgery (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: Anhui Provincial Hospital

New P2 trial • Surgery • Hematological Disorders • Hepatology • Thrombocytopenia